Arcturus Therapeutics announced positive interim Phase 2 results for ARCT-810. This investigational mRNA therapy targets Ornithine Transcarbamylase (OTC) deficiency, a rare genetic liver disorder.
The study demonstrated positive activity across key disease-related biomarkers. Treated patients achieved morning ammonia levels within the normal range.
Participants generally tolerated ARCT-810 well with no significant safety concerns reported. These findings support the continued clinical development of the mRNA-based treatment.